WORLDSymposium 2023 New Treatment Award

2023 New Treatment Award WORLDSymposium — Chester B. Whitley, Bill Sibold, Monica Kumar, Alaa Hamed, Shannon Resetich, Paul Hudson

WORLDSymposium strives to recognize important achievements in therapy for lysosomal diseases for treatments attaining regulatory approval. In recognition of achieving major milestones, WORLDSymposium reviews potential recipients for the New Treatment Award in 2023. This award honors “new treatments” that are viewed as providing value to patients with lysosomal diseases, and general acceptance as evidenced by approval by the U.S. Food and Drug Administration, European Medicines Agency, and other regulatory authorities.

The WORLDSymposium New Treatment Award for 2023 was presented to Sanofi for olipudase alfa-rpcp (Xenpozyme™) which provided clinical data meriting approval by the U.S. Food and Drug Administration (FDA) as the first disease-specific treatment of non-central nervous system (non-CNS) manifestations of acid sphingomyelinase deficiency (ASMD) in adult and pediatric patients.

Congratulations!

The New Treatment Award was presented on Saturday, February 25, 2023, at 7:45 AM EST, followed by a full day of Contemporary Forum abstract presentations.

Past Award Recipients:

2022 avalglucosidase alfa-ngpt (Nexviazyme®, Sanofi Genzyme)
2022 pabinafusp alfa (IZCARGO®, JCR Pharmaceuticals)
2018 cerliponase alfa (Brineura™, BioMarin)
2018 vestronidase alfa (MEPSEVII™, Ultragenyx)
2017 migalastat (Galafold™, Amicus)
2016 sebelipase alfa (Kanuma™, Alexion)
2015 eliglustat (Cerdelga™, Sanofi Genzyme)
2015 idursulfase beta (Hunterase®, GC Pharma)
2015 cysteamine bitartrate (PROCYSBI™, Horizon Therapeutics)
2015 elosulfase alfa (Vimizim®, BioMarin)